Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase 2, open-label, multi-center, 2-stage sequential cohort, dose escalation study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of subcutaneous SAR442501 in pediatric participants with Achondroplasia

This research is a Phase 2 study looking at a new medicine called SAR442501 for children and young people with achondroplasia, which is a condition affecting bone growth, leading to short stature. The medicine is given by injection under the skin. Researchers want to find out if this medicine is safe, how well people tolerate it, and how the body processes it. They are also exploring if it helps improve growth, affects bone development, and improves overall quality of life. This is an open-label study, meaning both participants and researchers will know who is receiving the medicine. It's a key step in understanding if this treatment could be helpful for those living with achondroplasia.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Sanofi-Aventis Recherche & Developpement

Enrolment target

Start

22 Sep 2023

Osteochondrodysplasia

What is this study about?

This study is focused on a genetic condition called achondroplasia, which is the most common form of dwarfism. It affects how bones grow, particularly in the arms and legs, leading to a shorter stature and sometimes other health challenges. Researchers are testing a new medicine, SAR442501, to see if it can help improve the growth of bones in children and young people with this condition. This medicine is given as an injection under the skin, similar to how some people might take insulin.

The main goal of this study is to check the safety of the new medicine. This means carefully watching for any side effects or unwanted reactions. The researchers will also look at how the body handles the medicine – how it's absorbed, distributed, used, and removed. Beyond safety, they're hoping to understand if the medicine has any positive effects on growth, such as changes in height or the proportions of different body parts. They will also look at whether it improves overall well-being and reduces pain.

This is a 'Phase 2' study, which means it's one of the earlier stages of testing a new medicine. It's designed to gather more detailed information about the medicine's effects and find the right dose before potentially moving on to larger studies. The aim is to develop a treatment that could make a real difference to the lives of people with achondroplasia.

Key takeaways

This study is testing a new medicine for achondroplasia.
The medicine is given as an injection under the skin.
The main goals are to check safety and see if it helps with growth.
It's an early-stage study, and more research will be needed.
You have the right to leave the study at any time.

Who may be eligible?

This study is looking for children and young people who have been diagnosed with achondroplasia. Importantly, the study is open to both boys and girls of all ages.

While the information provided doesn't list specific reasons why someone *wouldn't* be able to join, generally, studies have rules about other health conditions, medicines you might be taking, or if you've recently been part of another trial. These rules are in place to ensure the safety of everyone taking part and to make sure the study results are clear and accurate.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do you or your child have a confirmed diagnosis of achondroplasia?
Is the person considering joining a child or young person (all ages are welcome)?
Are you able to attend regular clinic visits?
Are you comfortable with the idea of injections and blood tests?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, the main part of it will involve receiving regular injections of the study medicine, SAR442501, under the skin. Your child will have these injections over a period of time, and both you and the study team will know that they are receiving the active medicine.

You can expect regular visits to the study clinic during this time. At these visits, the study team will carefully monitor your child's health and look for any side effects. They will also take measurements, such as height, arm and leg lengths, and head circumference. Blood tests will be taken to see how the medicine is working in the body and to check for any changes in bone markers. You may also be asked to fill out questionnaires about your child's quality of life, pain levels, and their development. The total duration of the study is not specified but will involve multiple assessments over time.

Potential risks and benefits

Participating in research always has potential benefits and risks. A potential benefit of this study is that the new medicine might help with bone growth and potentially improve other aspects of achondroplasia, though this is not guaranteed as it's an early-stage study. The possible risks include experiencing side effects from the medicine, which will be carefully monitored by the study team. There might also be some discomfort from injections and blood tests. It's very important to remember that joining a study is always your choice, and you are free to stop participating at any time, for any reason, without it affecting your usual medical care.

Locations (3)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Italy
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Unverified
Spain
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Unverified
Czechia

Common questions

What is achondroplasia?

Achondroplasia is a genetic condition that affects how bones grow, leading to shorter arms and legs, and shorter stature.

What does 'Phase 2 study' mean?

Phase 2 means it's an early-stage study, checking the safety of the new medicine and seeing if it has any positive effects, usually in a small group of people.

How is the medicine given?

The medicine, SAR442501, is given as an injection just under the skin.

Will we know if my child is getting the actual medicine?

Yes, this is an 'open-label' study, meaning everyone involved will know that the study medicine is being given.

What kind of tests will be done?

Tests will include checking for side effects, measuring growth, taking blood samples, and filling out questionnaires about health and well-being.